Abstract
LNP-mediated delivery of long coding RNA has been clinically validated for vaccines and gene editing. We have been developing a novel, synthetic, circular coding RNA platform (oRNA technology) which exhibits significant improvements in production, expression and formulation compared to mRNAs. Lacking the cap structure of mRNA, our oRNA technology uses a proprietary sequence-based IRES element to initiate protein translation in target cells. At the same time, ex vivo generated chimeric antigen receptor (CAR) T cell therapies have had tremendous success in treating hematologic malignancies, yet manufacturing, safety and efficacy challenges remain.
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